Catabasis Pharmaceuticals Reports Fourth Quarter and Full Year 2016 Financial Results and Recent Corporate Highlights
-- Part C of MoveDMD® Trial of Edasalonexent in Duchenne Muscular Dystrophy Ongoing --
-- Continued Advancement of Rare Disease Pipeline with CAT-5571, a Potential Treatment for Cystic Fibrosis, and CAT-4001, a Potential Treatment for Neurodegenerative Diseases --
“The recently reported safety, tolerability and plasma exposure data for
edasalonexent in patients with Duchenne muscular dystrophy from Part B
of the MoveDMD trial are reassuring, and Part C of the trial is
continuing on track,” said
Dr. Milne continued, “Looking forward in 2017, Part C of the MoveDMD trial will allow us to collect data for up to 48 weeks of treatment and provide important information about dose and activity with extended duration, as well as endpoints for possible future clinical trials of edasalonexent. Following additional data analysis from Part C, we will determine the next steps for edasalonexent in DMD. Catabasis is also moving the development of our rare disease pipeline forward, with advances in CAT-5571 for cystic fibrosis and CAT-4001 for neurodegenerative diseases including Friedreich’s ataxia and ALS.”
Recent and Upcoming Corporate Highlights
Edasalonexent (CAT-1004) and the MoveDMD Trial
- Announced top-line results from Part B of the MoveDMD trial of edasalonexent in January. The primary efficacy endpoint of MRI T2 was not met. The edasalonexent 100 mg/kg/day treatment group consistently showed numerical improvement versus placebo across multiple timed function tests and the North Star Ambulatory Assessment, although as expected the changes were not statistically significant. The 67 mg/kg/day treatment group had mixed results compared with both the 100 mg/kg/day treatment group and placebo, which in each case were not statistically significant.
-
Announced two publications about edasalonexent: Phase 1 clinical data
in the
Journal of Clinical Pharmacology and preclinical research in JCI Insight. - Ongoing open-label extension (Part C) of the MoveDMD trial in which patients continue on edasalonexent for 36 weeks following completion of Part B. Catabasis intends to report the results from Part C in 2017, with an interim update in Q2.
New Edasalonexent Updates
-
Upcoming presentation this month at the 2017
Muscular Dystrophy Association Scientific Conference , including: Part B data for the pediatric outcomes data collection instrument (PODCI) and muscle strength function-associated exploratory endpoints, which showed numerical improvement versus placebo for both edasalonexent treatment groups at 12 weeks as well as a review of previously released results from the MoveDMD trial. - In the Catabasis and Sarepta joint research collaboration, established to explore a combination drug treatment approach for DMD, increased dystrophin protein expression was seen with an exon-skip modality in combination with edasalonexent in the designated mouse model of DMD. The companies believe that these results warrant further research.
Additional Rare Disease Programs
-
Announced the publication of research on CAT-5571, a novel activator
of autophagy and potential oral treatment for cystic fibrosis (CF), in
the
Journal of Medicinal Chemistry . -
Presented preclinical data for CAT-5571 at the
North American Cystic Fibrosis Conference . CAT-5571, an activator of autophagy, in combination with lumacaftor/ivacaftor, enhanced cell-surface trafficking and function of CF transmembrane conductance regulator (CFTR) in bronchial epithelial cells from CF patients with the F508del mutation. Catabasis also presented that CAT-5571 enhanced the clearance of Pseudomonas aeruginosa infection in preclinical models of CF, irrespective of CFTR mutation status. - Ongoing preclinical activities exploring the potential of CAT-4001 in diseases such as amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia.
Corporate
-
Catabasis hosted its first Investor Day in
New York onNovember 17, 2016 , focused on its strategy in rare diseases and its pipeline, including edasalonexent and other programs. -
Catabasis had three recent executive team promotions:
Ted Hibben to Chief Business Officer,Andrew Nichols , Ph.D., to Chief Scientific Officer, and Angelika Fretzen, Ph.D., to Senior Vice President of Product Development.
Fourth Quarter and Full Year 2016 Financial Results
Cash Position: At
R&D Expenses: Research and development expenses were
G&A Expenses: General and administrative expenses were
Operating Loss: Loss from operations was
Net Loss: Net loss was
Conference Call and Webcast
Catabasis will host a conference
call and webcast at 4:30pm ET today to provide an update on corporate
developments and to discuss fourth quarter and full year 2016 financial
results.
Participant Toll-Free Dial-In Number: (877) 388-2733
Participant
International Dial-In Number: (541) 797-2984
Pass Code: 74976422
Please specify to the operator that you would like to join the “Catabasis Fourth Quarter and Full Year 2016 Results Call.”
Interested parties may access a live audio webcast of the conference call via the investor section of the Catabasis website, www.catabasis.com. Please connect to the Catabasis website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The webcast will be archived for 90 days.
About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is
an investigational oral small molecule that is being developed as a
potential disease-modifying therapy for all patients affected by
Duchenne muscular dystrophy (DMD or Duchenne), regardless of their
underlying mutation. Edasalonexent inhibits NF-kB, a protein that is
activated in Duchenne and drives inflammation and fibrosis, muscle
degeneration and suppresses muscle regeneration. In animal models of
DMD, edasalonexent produced beneficial effects in skeletal, including
diaphragm, and cardiac muscle and improved function. The
About CAT-5571
Catabasis is developing CAT-5571 as a
potential oral treatment for cystic fibrosis (CF) with potential effects
on both the cystic fibrosis transmembrane conductance regulator (CFTR)
and on the clearance of Pseudomonas aeruginosa. CAT-5571 is a
small molecule that activates autophagy, a process that maintains
cellular homeostasis and host defense mechanisms, and is known to be
impaired in CF. Catabasis has shown in preclinical studies that
CAT-5571, in combination with lumacaftor/ivacaftor, enhances
cell-surface trafficking and function of CFTR with the F508del mutation.
Catabasis has also shown that CAT-5571 enhances the clearance of P.
aeruginosa infection in preclinical models of CF, irrespective of
CFTR mutation status.
About CAT-4001
Catabasis is developing CAT-4001 as a
potential treatment for neurodegenerative diseases such as Friedreich’s
ataxia (FA) and amyotrophic lateral sclerosis (ALS). CAT-4001 is a small
molecule that activates Nrf2 and inhibits NF-kB, two pathways that have
been implicated in FA and ALS. Catabasis has shown that CAT-4001
modulates the Nrf2 and NF-kB pathways in both cellular assays and animal
models.
About Catabasis
At
Forward Looking Statements
Any statements in this press
release about future expectations, plans and prospects for the Company,
including statements about future clinical trial plans and other
statements containing the words “believes,” “anticipates,” “plans,”
“expects,” “may” and similar expressions, constitute forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995. Actual results may differ materially from those
indicated by such forward-looking statements as a result of various
important factors, including: uncertainties inherent in the initiation
and completion of preclinical studies and clinical trials and clinical
development of the Company’s product candidates; availability and timing
of results from preclinical studies and clinical trials; whether interim
results from a clinical trial will be predictive of the final results of
the trial or the results of future trials; expectations for regulatory
approvals to conduct trials or to market products; availability of
funding sufficient for the Company’s foreseeable and unforeseeable
operating expenses and capital expenditure requirements; other matters
that could affect the availability or commercial potential of the
Company’s product candidates; and general economic and market conditions
and other factors discussed in the “Risk Factors” section of the
Company’s Annual Report on Form 10-K for the year ended
Catabasis Pharmaceuticals, Inc. |
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Condensed Consolidated Statements of Operations |
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(In thousands, except share and per share data) |
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(Unaudited) |
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Three Months Ended December 31, | Year Ended December 31, | |||||||||||||||||||||
2016 | 2015 | 2016 | 2015 | |||||||||||||||||||
Operating expenses: | ||||||||||||||||||||||
Research and development | $ | 6,260 | $ | 6,670 | $ | 25,450 | $ | 23,030 | ||||||||||||||
General and administrative | 2,413 | 2,663 |
|
10,108 |
|
8,629 |
||||||||||||||||
Total operating expenses |
8,673 | 9,333 |
|
35,558 |
|
31,659 |
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Loss from operations | (8,673 | ) | (9,333 | ) |
|
|
(35,558) |
|
|
(31,659) |
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Other (expense) income: | ||||||||||||||||||||||
Interest expense | (175 | ) | (269 | ) |
|
|
(837) |
|
|
(978) |
||||||||||||
Interest and investment income | 59 | - |
|
242 |
|
- |
||||||||||||||||
Other income, net | 11 | (4 | ) |
|
93 |
|
7 |
|||||||||||||||
Total other expense, net | (105 | ) | (273 | ) |
|
|
(502) |
|
|
(971) |
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Net loss | $ | (8,778 | ) | $ | (9,606 | ) | $ | (36,060) | $ | (32,630) | ||||||||||||
Net loss per share - basic and diluted | $ | (0.47 | ) | $ | (0.63 | ) | $ | (2.22 | ) | $ | (4.06 | ) | ||||||||||
Weighted-average common shares |
18,699,480 | 15,298,810 |
|
16,230,190 |
|
8,041,948 |
Catabasis Pharmaceuticals, Inc. |
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Condensed Consolidated Balance Sheets |
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(In thousands) |
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(Unaudited) |
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As of December 31, | |||||||||
2016 | 2015 | ||||||||
Assets | |||||||||
Cash and cash equivalents | $ | 23,596 | $ | 62,780 | |||||
Available-for-sale securities | 14,931 | - | |||||||
Total assets | 40,209 | 64,169 | |||||||
Liabilities and stockholders’ equity | |||||||||
Current portion of notes payable, net of discount | 3,243 | 3,173 | |||||||
Notes payable, net of current portion and discount | 2,479 | 5,720 | |||||||
Total liabilities | 11,123 | 13,676 | |||||||
Total stockholders’ equity | $ | 29,086 | $ | 50,493 |
Catabasis Pharmaceuticals, Inc. |
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Condensed Consolidated Statements of Cash Flows |
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(In thousands) |
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(Unaudited) |
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Year Ended December 31, | ||||||||||
2016 | 2015 | |||||||||
Net cash used in operating activities | $ | (32,858 | ) | $ | (29,793 | ) | ||||
Net cash used in investing activities | (15,490 | ) | (421 | ) | ||||||
Net cash provided by financing activities | 9,164 | 78,326 | ||||||||
Net (decrease) increase in cash and cash equivalents | $ | (39,184 | ) | $ | 48,112 |
View source version on businesswire.com: http://www.businesswire.com/news/home/20170316006156/en/
Source:
Corporate and Media Contact
Catabasis
Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com